News FDA Clears Phase 3 Trial Testing EB-101 Cell Therapy for Severe Blistering Skin Disorder

News Gene Therapy Takes Center Stage at 2019 NORD Summit

News HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

News New COL7A1 Gene Mutation Found in Rare DEB Subtype, Study Shows

News Precautions Can Help Ensure Safe Pregnancy, Delivery in Women With EB Simplex, Study Reports

News Rare Disease Film Festival Highlights Patient and Researcher Unity

News Cannabinoid-based INM-755 May Soon be Tested in Phase 1 Trial in Netherlands

News Gene Therapy B-VEC Is Safe, Leads to Sustained Wound Healing in RDEB Patients, Phase 1/2 Trial Shows

News EB-101 Cell Therapy Safely Heals Wounds in Adults with RDEB, Early Trial Results Show

News Serlopitant May Reduce Itching in EB, But More Research Is Needed

News New Guidelines for Foot Care in EB Stress Use of Podiatrists, Proper Footwear

News FDA Halts Phase 3 Trial of Abeona’s EB-101 for RDEB Due to Transport Concerns

News NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

News New Gene Editing Technique Shown to Correct COL7A1 Gene in RDEB Cells

News DAAC Dressing Closes EB Wounds Faster Than Standard Saline-Antibiotic One, Study Says

News Cell Therapy for RDEB Needs Collagen VII Repair in Two Types Skin Cells to Achieve Stability, Study Finds

News ‘Disorder’ Film Festival Offers Look at Siblings Touched by Rare Diseases

News First Patient Dosed in Phase 2 Trial of RGN-137 Achieves Complete Wound Healing, RegeneRx Announces

News At NY Genome Center, Legal Expert Presents Ethical Dilemmas in Gene Editing

News Injecting Engineered Cells May Prevent Blistering in RDEB, Study Indicates

News Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

News Amryt Releases Design of Pivotal Trial Evaluating Oleogel-S10 for EB Patients of All Ages

News Aegle Wins Debra’s Partners in Progress Award, Plans to Soon Open Clinical Trial in DEB Patients

News Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card

News Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate

News Gene-modified Skin Cells Show Promise for RDEB in Phase 1 Trial

News Transplants Using Patients’ Own Skin May Be Safe, Effective Treatment for RDEB, Trial Suggests

News European Initiative Targets Diagnosis, Treatment of Rare Diseases

News FDA Releases Guidelines to Push Industry Development of New Therapies

News Macedonian Gaucher Activist Publicizes Plight of Rare Disease Patients

News World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

News KB103 Continues to Show Promise in Treating DEB, Receives RMAT Designation from FDA

News NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

News Vital Signs Monitor May Lower Risk of Skin Injury in NICU Babies and EB Patients

News Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

News ‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

News EB-101, Potential Cell Therapy for RDEB, Seen to Lead to Sustained Wound Healing in Trial

News RDEB Gene Therapy Candidate FCX-007 Given FDA Regenerative Medicine Advanced Therapy Status

News First Patient Dosed in Phase 2 Trial Testing RGN-137 for Epidermolysis Bulllosa

News Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

News Gene Therapy Using CRISPR-Cas9 Shows Promise as Safer, More Efficient Method to Treat RDEB, Preliminary Study Shows

News ProQR Creates New Company, Wings Therapeutics, to Assume Development of QR-313 for Dystrophic EB

News Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

News Castle Creek, Fibrocell Teaming Up to Advance FCX-007 for Recessive Dystrophic Epidermolysis Bullosa

News PTR-01 Granted FDA’s Fast Track Status for Treatment of Recessive Dystrophic Epidermolysis Bullosa

News Approval of Off-the-Shelf Stem Cell Therapy to Treat Epidermolysis Bullosa Sought in Japan, JCR Pharmaceuticals Says

News Investigational Gene Therapy KB103 Receives Priority Designation in Europe for Dystrophic Epidermolysis Bullosa

News Third Annual ‘Believe in Brady’ Fundraiser to Benefit EB Research Partnership

News Rigosertib May Be Effective, Safe for Squamous Cell Carcinoma in RDEB Patients, Study Suggests

News RDEB Patients at Higher Risk of Osteoporosis, Fractures, Study Suggests

News JEB Involving Kidneys, Lungs Should be Classified as Mixed Form of Disease, Case Report Suggests

News First Patients Dosed in Phase 1/2 Trial Testing PTR-01 for Recessive Dystrophic Epidermolysis Bullosa

News Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn

News Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist

News NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research

News Prognosis Good for Pediatric Autoimmune Blistering Diseases, but Long-Term Follow-up Needed, Study Says

News WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

News Care, Management of EB Simplex Infant During Eye Surgery Detailed in Case Report

News Vienna to Host RARE2019 Meeting on Rare Diseases

News Mechanism Underlying Fibrosis in RDEB Could Be New Therapeutic Target

News Blocking Inflammatory Pathway Reduces Blisters in EB Simplex Generalized Severe Patients

News Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

News Steroid-induced Diabetes in EBA Patient Shows Importance of Follow-up Care

News Cannabinoids Could Help Manage EB-related Pain, Study Suggests

News Next-generation DNA Sequencing Efficiently Identifies Novel Mutation Causing Junctional EB in Newborn

News Clinical Diagnosis More Accurate Than Skin Biopsy in Inherited EB Patients, Study Reports

News Severe EB Requires Multidisciplinary Approach, Case Report Suggests

News Decorin Extends Survival, Eases Skin Fibrosis in Mouse Model of RDEB

News Krystal Biotech Completes New Plant Construction to Support KB103 Development in Treating DEB

News Independent Committee Supports Additional Patient Enrollment in Phase 3 Trial of AP101 in EB Patients

News Researchers Create Dressing Glove for EB Based on Patients’ Feedback

News Diagnostic Testing More Common in Most Severe EB Types, Study Reports

News Collagen XVII Mutation Behind Skin Blistering Seen in Mild JEB, Study Confirms

News Four Patients Treated in Phase 2 Trial Testing Topical Gene Therapy, KB103, for Wound Healing

News Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

News EB Groups Give Fibrocell $900,000 to Advance Gene Therapy in Clinical Trials

News Phase 3 Trial of RDEB Therapy EB-101 to Start in 2019, Abeona Therapeutics Says

News TXA127 Enters Development Program as Potential Treatment for EB

News Diacerein Ointment Could Be Safe Treatment for Generalized Severe EBS, Study Suggests

News Intravenous Immunoglobulin and Rituximab Most Effective EBA Treatments, Study Reports

News RegeneRX, GtreeBNT Provide Update on RGN-137 for Treatment of Epidermolysis Bullosa

News #NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

News FDA Advises Fibrocell on Phase 3 Trial Design for FCX-007, Potential RDEB Therapy

News TWi Biotechnology Enrolls First Patient in Phase 2 Trial Testing AC-203 for EB Treatment

News DNA Sequencing Technique Identifies Two New Mutations in Patient with Simplex and Junctional EB

News First RDEB Adult Patients Do Well in Clinical Trial with Gene Therapy KB103

News Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

News Gene Editing Corrects Mutation Causing Recessive Dystrophic Epidermolysis Bullosa, Study Reports

News Calcipotriol Ointment Seen to Improve Wound Healing in RDEB Patient, Study Finds

News Fibrocell Awarded $1.4M from FDA to Advance FCX-007 in Clinical Trials for RDEB

News #NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

News Dad Who Lost Baby to EB Nears Final Stretch in ‘Big Ride’ to Raise Money to Combat Disease

News Specific Enzymes Cause Mutations That Fuel Aggressive Skin Cancer in Patients With RDEB, Study Shows

News Amryt’s AP101 for Treating EB Receives IND Clearance from FDA

News RDEB Is Main Suspect Behind SCC, a Rare Form of Skin Cancer, Study Shows

News 2 EB Research Groups Based Continents Apart Partner to Advance New Treatments

News Case Report Describes Successful Treatment of JEB with Terrasil Ointment

News Austrian Epidermolysis Bullosa Patient Inspires Dad to Establish World’s First ‘EB House’

News FDA Grants Fast Track Status to Castle Creek’s CCP-020 for Treatment of EBS

News Joint Venture Plans to Advance Potential EB Therapy RGN-137 into Phase 3 Trial